LA JOLLA, Calif., April 15, 2016 /PRNewswire/ -- Regulus
Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company
leading the discovery and development of innovative medicines
targeting microRNAs, today announced additional interim results
from one of the company's ongoing Phase II studies of RG-101 for
the treatment of Hepatitis C Virus infection (HCV) during an oral
presentation at the International Liver Congress 2016 (ILC 2016)
taking place April 13-17 in
Barcelona, Spain. The study was
designed to evaluate a shortened, four-week treatment regimen
containing a subcutaneous administration of 2 mg/kg of RG-101 at
Day 1 and Day 29, in combination with 4 weeks of once/daily
approved anti-viral agents Harvoni®, Olysio®, or Daklinza™.
The study enrolled 79 treatment naïve genotype 1 and 4 HCV patients
(Harvoni® arm, n=27, Olysio® arm, n=27, Daklinza™ arm,
n=25).
Today, Regulus reported longer-term follow up results across all
arms of the ongoing study:
Follow-Up After
2nd
Dose of RG-101
|
RG-101
+
Harvoni®
|
RG-101
+
Olysio®
|
RG-101
+
Daklinza™
|
Week
8
|
21/21
(100%)
|
21/21
(100%)
|
20/22*
(90.9%)
|
Week
12
|
14/14
(100%)
|
14/15*
(93.3%)
|
12/12
(100%)
|
Week
16
|
9/9 (100%)
|
8/9*
(88.9%)
|
9/9
(100%)
|
Week
20
|
2/2
(100%)
|
2/2
(100%)
|
2/2
(100%)
|
Week
24
|
1/1
(100%)
|
2/2
(100%)
|
-- (--)
|
Response defined as HCV RNA viral load below LLOQ using
RealTime HCV Assay (Abbott) with LLOQ = 12
*Includes one relapse patient in the Olysio arm (week
12) and one relapse patient in the Daklinza arm (week 8)
"We believe the data reported at the ILC meeting further
demonstrate the clinical utility of RG-101 to shorten oral HCV
treatment regimens to just four weeks," said Paul Grint, M.D., President and CEO of
Regulus. "With interim data now through 24 weeks of
follow-up, the consistent trend in efficacy and safety is
encouraging, which supports the potential of RG-101 to become a
backbone agent in combination with all classes of oral therapies.
Throughout the year, we look forward to obtaining more data from
multiple studies across our broad Phase 2 development program."
To date, RG-101 has been generally well tolerated with the
majority of adverse events considered mild or moderate, and with no
study discontinuations. Changes in pharmacodynamic markers
are indicative of effective target engagement and consistent with
the company's prior experience with miR-122 inhibition. The
primary endpoint analysis (12 week follow up) for all 79 patients
in the study is anticipated to be reported in late Q2 2016.
The investigator slides presented at the ILC 2016 meeting are
available on the investor relations page of Regulus' website at
www.regulusrx.com.
Conference Call & Webcast Information
Today at 8:00 a.m. EDT, Regulus
will host a conference call and webcast to discuss the ILC 2016
interim results. A live webcast of the call will be available
online at www.regulusrx.com. To access the call, please dial
(877) 257-8599 (domestic) or (970) 315-0459 (international) and
refer to conference ID 89822720. To access the telephone
replay of the call, dial (855) 859-2056 (domestic) or (404)
537-3406 (international), passcode 89822720. The webcast and
telephone replay will be archived on the company's website
following the call.
About Hepatitis C Virus Infection (HCV)
Hepatitis C is a result of a hepatocyte specific infection
induced by the virus known as HCV. Chronic HCV may lead to
significant liver disease, including chronic active hepatitis,
cirrhosis, and hepatocellular carcinoma. Up to 185 million people
are chronically infected with HCV worldwide, and more than 500,000
people die from HCV annually. The CDC estimates that there
are currently approximately 3.5 million persons infected with HCV
in the United States. HCV shows significant genetic variation
in worldwide populations due to its frequent rates of mutation and
rapid evolution. There are six genotypes of HCV, with several
subtypes within each genotype, which vary in prevalence across the
different regions of the world. The response to treatment varies
from individual to individual underscoring the inadequacy of
existing therapies and highlights the need for combination
therapies that not only target the virus but endogenous host
factors as well, such as microRNA-122 (miR-122). Regulus believes
that its miR-122 antagonist, RG-101, may be a useful agent in
emerging combination regimens to address difficult-to-treat
genotypes and to potentially expand upon the current therapies
available to clinicians treating HCV patients.
About RG-101 for HCV
RG-101 is Regulus' wholly-owned, GalNAc-conjugated anti-miR
targeting miR-122, which the HCV virus uses to replicate. Therapies
that interfere with miR-122 could inhibit viral replication, acting
earlier in the viral life cycle than currently approved oral
agents. In a completed Phase I human proof-of-concept study,
Regulus demonstrated that treatment with a single subcutaneous dose
of RG-101 as monotherapy resulted in significant and sustained
viral load reductions in all treated HCV patients, including
patients with difficult to treat genotypes, various liver fibrosis
status and those who have experienced viral relapse after a prior
IFN-containing regimen.
Regulus has reported favorable interim data from an ongoing
Phase II study evaluating the combination of RG-101 with multiple
approved DAAs positioning RG-101 for both front-line and
second-line commercial opportunities. Patients received a single
subcutaneous injection of 2 mg/kg of RG-101 on Day 1, followed by
28 days of a once daily oral DAA (Harvoni®, Olysio®, or Daklinza™),
followed by an additional subcutaneous injection of 2 mg/kg of
RG-101 on Day 29. Regulus is planning to report primary
endpoint results at 12 weeks following conclusion of treatment in
late Q2 2016.
In collaboration with GSK, Regulus recently initiated a Phase II
study evaluating the combination of RG-101 and GSK2878175, a
non-nucleoside NS5B polymerase inhibitor, in treatment-naïve
patients chronically infected with HCV genotypes 1 and 3.
Additionally, enrollment is nearly complete in a multi-center, open
label, non-randomized Phase I study to compare the safety,
tolerability, pharmacokinetics, and pharmacodynamics of 2 mg/kg of
RG-101 in subjects with severe renal insufficiency or end-stage
renal disease (ESRD) to healthy control subjects, and further
explore RG-101 in hepatitis C infected subjects with severe renal
insufficiency or ESRD. Regulus anticipates reporting safety and
efficacy data from the HCV/severe renal impairment or ESRD arm in
the second half of 2016.
About microRNAs
The discovery of microRNAs in humans during the last decade is
one of the most exciting scientific breakthroughs in recent
history. microRNAs are small RNA molecules, typically 20 to
25 nucleotides in length, that do not encode proteins but instead
regulate gene expression. More than 800 microRNAs have been
identified in the human genome, and over two-thirds of all human
genes are believed to be regulated by microRNAs. A single
microRNA can regulate entire networks of genes. As such, these
molecules are considered master regulators of the human
genome. microRNA expression, or function, has been shown to
be significantly altered or dysregulated in many disease states,
including oncology, fibrosis, metabolic diseases,
immune-inflammatory diseases and HCV. Targeting microRNAs with
anti-miRs, chemically modified, single-stranded oligonucleotides,
offers a unique approach to treating disease by modulating entire
biological pathways and may become a new and major class of drugs
with broad therapeutic application.
About Regulus
Regulus Therapeutics Inc. (NASDAQ: RGLS) is a
biopharmaceutical company leading the discovery and development of
innovative medicines targeting microRNAs. Regulus has
leveraged its oligonucleotide drug discovery and development
expertise to develop a well-balanced microRNA therapeutics pipeline
complemented by a maturing
microMarkersSM biomarkers platform and a rich
intellectual property estate to retain its leadership in the
microRNA field. Regulus is developing RG-101, a
GalNAc-conjugated anti-miR targeting microRNA-122 for the treatment
of chronic hepatitis C virus infection, and RG-012, an anti-miR
targeting microRNA-21 for the treatment of Alport syndrome, a
life-threatening kidney disease driven by genetic mutations with no
approved therapy. In addition, RG-125, a GalNAc-conjugated
anti-miR targeting microRNA-103/107 for the treatment of NASH in
patients with type 2 diabetes/pre-diabetes, has entered Phase I
clinical development through its strategic alliance with
AstraZeneca. Regulus is also advancing several programs
toward clinical development in orphan disease indications, oncology
and fibrosis. Regulus' commitment to innovation has resulted
in multiple peer-reviewed publications in notable scientific
journals and has resulted in the formation of strategic alliances
with AstraZeneca and Sanofi. Regulus maintains its corporate
headquarters in La Jolla, CA. For more information,
please visit http://www.regulusrx.com.
Forward-Looking Statements
Statements contained in this press release regarding matters
that are not historical facts are "forward-looking statements"
within the meaning of the Private Securities Litigation Reform Act
of 1995, including statements associated with the expected ability
of Regulus to undertake certain activities and accomplish certain
goals (including with respect to development and other activities
related to RG-101), the projected timeline of clinical development
activities, and expectations regarding future therapeutic and
commercial potential of Regulus' business plans, technologies and
intellectual property related to microRNA therapeutics and
biomarkers being discovered and developed by Regulus. Because
such statements are subject to risks and uncertainties, actual
results may differ materially from those expressed or implied by
such forward-looking statements. Words such as "believes,"
"anticipates," "plans," "expects," "intends," "will," "goal,"
"potential" and similar expressions are intended to identify
forward-looking statements. These forward-looking statements are
based upon Regulus' current expectations and involve assumptions
that may never materialize or may prove to be incorrect.
Actual results and the timing of events could differ materially
from those anticipated in such forward-looking statements as a
result of various risks and uncertainties, which include, without
limitation, risks associated with the process of discovering,
developing and commercializing drugs that are safe and effective
for use as human therapeutics, and in the endeavor of building a
business around such drugs. These and other risks concerning
Regulus' financial position and programs are described in
additional detail in Regulus filings with the Securities and
Exchange Commission. All forward-looking statements contained
in this press release speak only as of the date on which they were
made. Regulus undertakes no obligation to update such statements to
reflect events that occur or circumstances that exist after the
date on which they were made.
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